Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes. (2020)
Abstract
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Bibliographic Information
Digital Object Identifier: http://dx.doi.org/10.1093/hmg/ddz317
PubMed Identifier: 31919491
Publication URI: http://europepmc.org/abstract/MED/31919491
Type: Journal Article/Review
Volume: 29
Parent Publication: Human molecular genetics
Issue: 12
ISSN: 0964-6906